Clinical Trials Logo
  1. Home
  2. Spinal Muscular Atrophy Type 3
  3. NCT06421831

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

Spinal Muscular Atrophy Type 3 Clinical Trial

Official title:
A Multi-center, Open Label, Single-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

Clinical Trial Summary

The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.

Clinical Trial Description

The purpose of this trial is to evaluate safety and efficacy of gene therapy drug GC101 in SMA 3 patients. Open-label, dose-escalation clinical trials of GC101 will be conducted in multiple centers in China. GC101 will be administrated intrathecally. Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will. Patients will be tested at baseline and followed up at various time points. The primary analysis for efficacy will be assessed at 12 months after treatment with GC101 on the changes from baseline HFMSE (Hammersmith Functional Motor Scale Expanded) and RULM（Revised Upper Limb Module) scores for patients of age ≥ 6 years old. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06421831
Study type Interventional
Source GeneCradle Inc
Contact GeneCradle, Inc China
Phone +8613501380583
Email ind@bj-genecradle.com
Status Recruiting
Phase Phase 1/Phase 2
Start date May 10, 2024
Completion date December 2028
View Details
See also
  Status Clinical Trial Phase
Not yet recruiting NCT06300996 - Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb N/A
Completed NCT04193085 - Wearable Technology to Assess Gait Function in SMA and DMD
Completed NCT02895789 - Oxidative Capacity and Exercise Tolerance in Ambulatory SMA
Terminated NCT03819660 - Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 Phase 2
Recruiting NCT05272969 - Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease
Recruiting NCT05544994 - The Effect of Aerobic Exercise Training in Patients With Type III Spinal Muscular Atrophy N/A
Completed NCT02227823 - Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3 Phase 2
Active, not recruiting NCT05156320 - Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam Phase 3
Recruiting NCT03709784 - Spinraza in Adult Spinal Muscular Atrophy
Terminated NCT03056144 - Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy N/A
Recruiting NCT05430113 - Spinal Cord Stimulation in Spinal Muscular Atrophy N/A
Completed NCT03921528 - An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy Phase 2
Active, not recruiting NCT05626855 - Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX Phase 3
Completed NCT04907162 - Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders